Gaming the regulatory apparatus to advance the human condition

Vanna Belton from Baltimore has been blind for more than five years, but after undergoing surgery where stem cells extracted from her bone marrow were injected into her right eye’s retina and left eye’s optic nerve, she has regained some of her sight.

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The doctor who performed the stem cell treatment, ophthalmologist Jeffrey N. Weiss, cut so many corners to get her and 277 other patients into surgery, even he can’t explain why it works.

“Weiss is not following the usual steps of clinical studies,” Meredith Cohn reports for the Baltimore Sun. “Among other things, he didn’t test his treatment theories first on lab animals or using computer models, or randomise his trials by using either stem cells or placebos in study participants. He didn’t test the procedure for safety on a small group before moving to a larger trial.”

Essentially, the ‘traditional route’ of lab-to-animals-to-clinical-trials takes too long, Weiss says, so he figured out how to game the system and start testing his new treatment on his own terms.

All he had to do was register his human trials with the US National Institutes of Health (NIH), which usually requires approval from the Food and Drug Administration (FDA) before any new or established drugs can be tested on humans.

The catch is that stem cells aren’t classified as a drug – they’re extracted from a patient’s body, go through very little processing, and are used on that same patient, so Weiss was able to bypass FDA approval to register his NIH trials. He did need approval and oversight from an ethics review panel, and got his from the International Cellular Medicine Society, an independent, pro-stem cell therapy group based in Nevada.

So Weiss, who is practicing independently of any university or research institution, is now free to charge around US$20,000 per patient to perform his stem cell treatment, and offers no promises of a cure to his patients.

But according to Cohn, Weiss reports that 60 percent of his 278 patients – who have gone blind because of diseases such as macular degeneration and glaucoma – have regained some sight following the procedure. He’s published a case study on Belton in the journal Neural Regeneration Research.